Gene Therapy Briefs

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Advanced Cell Technology (ACT) said its ability to carry out the first human embryonic stem cell (hESC) trial in Europe won’t be affected by the Court of Justice of the European Union’s decision that stem cell research based on the destruction of human embryos cannot be patented (see Legal Affairs on page 10 for more information about this decision).“It should have minimal impact on our commercial efforts in the EU,” because ACT’s European programs employ a single-cell blastomere technique that allows for deriving hESC lines without destroying embryos, Gary Rabin, ACT’s chairman and CEO, said. ACT has begun treating patients as part of a Phase I/II clinical trial for Stargardt’s Macular Dystrophy using retinal pigment epithelium derived from hESCs.

Amsterdam Molecular Therapeutics (AMT) laid off half its workforce, down to 45 employees, and suspended its development of Glybera for lipoprotein lipase deficiency, and its Duchenne muscular dystrophy program. The moves followed rejection of a marketing authorization for Glybera by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP). Glybera’s benefits did not outweigh its risks despite the drug’s potential value, CHMP concluded.“In our industry, the difference between success and failure is often tiny. And in this particular case I would say it was just one voice in the CHMP,” AMT CEO Jorn Aldag told analysts. CHMP prevailed over EMA’s Committee for Advanced Therapies, which said Glybera was approvable subject to post-marketing studies.

Oxford BioMedica and the Mayo Clinic, Rochester have teamed up for an R&D collaboration that aims to develop a gene therapy for treating chronic glaucoma. The partners will carry out preclinical work to test the feasibility of using a therapeutic based on Oxford BioMedica’s LentiVector® gene-delivery system expressing a COX-2 gene and a PGF-2alpha receptor gene, for use in reducing intraocular pressure.

Sanofi, which completed a $20.1 billion acquisition of Genzyme in April, will restructure R&D operations by consolidating into a single new Genzyme R&D Center the old Genzyme’s rare disease program, and the multiple sclerosis programs of both predecessor companies. Sanofi will absorb the global operations of Genzyme’s former biosurgery, oncology, and renal divisions.

In a trial led by Oxford University in collaboration with Imperial College London, a 63-year-old arbitration lawyer from Bristol, U.K., is the first patient to be treated with a gene therapy technique for an incurable form of blindness. The technique is designed to save his sight by reversing his Choroideraemia, a rare genetic blindness.Jonathan Wyatt is the first of 12 patients to be treated with the gene therapy in one eye. The other eye will act as a control, but will also be treated if the therapy proves successful over the next two years.

Geron presented Phase I data showing that GRNOPC1 “has been well tolerated with no serious adverse events” after the lowest intended dose of the human embryonic stem cell-based therapy was given to four patients with complete thoracic spinal cord injuries.GRNOPC1 was administered between 7 and 14 days after injury. Low-dose tacrolimus was given for temporary immune-suppression from time of injection for 46 days, when the dose was tapered and withdrawn completely at 60 days. A few mild adverse events related to tacrolimus were reported, but with no evidence of cavitation in the spinal cord at the injury sites on MRI, no unexpected neurological changes, and no evidence of immune responses to GRNOPC1.

Launching in 2012: Human Gene Therapy Methods

The field of gene therapy is evolving and and holds great promise for its applications in treating human disease. Human Gene Therapy, the premier in the field, is launching Human Gene Therapy Methods to complement the flagship publication. HGT Methods answers the growing need for a central forum for nurturing, promoting, and advancing new technologies and methods that will ultimately pave the way for product development. The Editor in Chief is James M. Wilson, M.D., Ph.D.